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Showing 1–24 of 506 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 3 Interventional Results available

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

NCT04759131
View directory record Official record
Conditions
Hemophilia A
Interventions
efanesoctocog alfa (BIVV001)
Drug
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Jul 25, 2026, 11:23 AM EDT
Terminated Phase 3 Interventional

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03917719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Edasalonexent
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 22, 2020 · Synced Jul 25, 2026, 11:23 AM EDT
Not listed No phase listed Observational

Systemic Therapies for Pediatric Atopic Dermatitis

NCT01447381
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Not listed
Lead sponsor
Rady Children's Hospital, San Diego
Other
Eligibility
2 Years to 17 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2021
U.S. locations
3
States / cities
San Diego, California • San Francisco, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 4, 2012 · Synced Jul 25, 2026, 11:23 AM EDT
Terminated Phase 3 Interventional Results available

Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease

NCT03615924
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Ticagrelor, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
2 Years to 17 Years
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
7
States / cities
Fort Lauderdale, Florida • Miami, Florida • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Jul 25, 2026, 11:23 AM EDT
Suspended Not applicable Interventional Accepts healthy volunteers

Artisan Aphakia Lens for the Correction of Aphakia in Children

NCT01547442
View directory record Official record
Conditions
Aphakia
Interventions
Artisan Aphakia Intraocular Lens
Device
Lead sponsor
Ophtec USA
Industry
Eligibility
2 Years to 21 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2025
U.S. locations
18
States / cities
Phoenix, Arizona • Los Angeles, California • Indianapolis, Indiana + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2024 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Talabostat Combined With Temozolomide or Carboplatin in Treating Young Patients With Relapsed or Refractory Brain Tumors or Other Solid Tumors

NCT00303940
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
carboplatin, talabostat mesylate, temozolomide, pharmacological study
Drug · Other
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
2 Years to 18 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 14, 2012 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Not applicable Interventional Results available

Care Plans for Cancer Predisposition

NCT05736497
View directory record Official record
Conditions
Genetic Predisposition
Interventions
Digital Care Plans
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
12 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Jul 25, 2026, 11:23 AM EDT
Not listed Phase 1Phase 2 Interventional

Single Ascending Dose Study in Participants With LCA10

NCT03872479
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2022 · Synced Jul 25, 2026, 11:23 AM EDT
Not listed Phase 3 Interventional

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

NCT04860960
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin, Placebo
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2024 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Effect of Different Skin Creams on TEWL

NCT03663673
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
EpiCeram, Aveeno Daily Moisturising Sheer Hydration Lotion®
Drug
Lead sponsor
Stanford University
Other
Eligibility
1 Day to 40 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Mountain View, California
Source: ClinicalTrials.gov public record
Updated Sep 7, 2021 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 2 Interventional Results available

Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function

NCT05001269
View directory record Official record
Conditions
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
Interventions
nedosiran
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Up to 11 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 25, 2026, 11:23 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

NCT04806451
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Crinecerfont, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
2 Years to 17 Years
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
22
States / cities
Birmingham, Alabama • Los Angeles, California • Orange, California + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2025 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 4 Interventional Results available

Cutivate Lotion HPA Axis Pediatric Study

NCT00546000
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Fluticasone propionate 0.05% lotion
Drug
Lead sponsor
Fougera Pharmaceuticals Inc.
Industry
Eligibility
3 Months to 12 Months
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
10
States / cities
Poway, California • San Diego, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced Jul 25, 2026, 11:23 AM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359
View directory record Official record
Conditions
Hypophosphatasia
Interventions
ALXN1850, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Baltimore, Maryland • Kansas City, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Carisbamate in Adult & Pediatric Subjects With Lennox-Gastaut Syndrome

NCT03731715
View directory record Official record
Conditions
Lennox Gastaut Syndrome
Interventions
Carisbamate
Drug
Lead sponsor
SK Life Science, Inc.
Industry
Eligibility
2 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
7
States / cities
Baltimore, Maryland • Lebanon, New Hampshire • Durham, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated May 29, 2023 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

NCT03096080
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Recessive
Interventions
Tesevatinib
Drug
Lead sponsor
Kadmon Corporation, LLC
Industry
Eligibility
5 Years to 12 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 10, 2022 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 2 Interventional

A Study in Pediatric Patients With Cystic Fibrosis Lung Disease

NCT00130182
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
denufosol tetrasodium (INS37217)
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
5 Years to 7 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
3
States / cities
Denver, Colorado • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 21, 2013 · Synced Jul 25, 2026, 11:23 AM EDT
Terminated Phase 1 Interventional

Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I)

NCT03396575
View directory record Official record
Conditions
Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma
Interventions
TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning, Dose-Intensified TMZ, Td vaccine, Autologous Hematopoietic Stem Cells (HSC)
Biological · Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Years to 30 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia

NCT01506544
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
2 Years to 17 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
6
States / cities
Birmingham, Alabama • Chicago, Illinois • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 11, 2014 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)

NCT01581593
View directory record Official record
Conditions
Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
Interventions
Kedrion IVIG 10%
Biological
Lead sponsor
Kedrion S.p.A.
Industry
Eligibility
2 Years to 70 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
12
States / cities
Little Rock, Arkansas • North Palm Beach, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 2Phase 3 Interventional

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

NCT04902781
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007, Placebo
Drug · Other
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Atlanta, Georgia • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Jul 25, 2026, 11:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)

NCT05312177
View directory record Official record
Conditions
Down Syndrome, Congenital Heart Disease
Interventions
Stanford Binet Intelligence Scales, Fifth Edition, Vineland Adaptive Behavior Scales Third Edition, Peabody Picture Vocabulary Test, Fifth Edition, Expressive Vocabulary Test, Third Edition, Leiter International Performance Scale, Third Edition, Social Communication Questionnaire, Current Form, Aberrant Behavior Checklist, Repetitive Behavior Scale, Revised
Other
Lead sponsor
Carelon Research
Other
Eligibility
60 Months to 155 Months
Enrollment
273 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
13
States / cities
Wilmington, Delaware • Washington D.C., District of Columbia • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 4 Interventional Results available

Dexmedetomidine and Propofol for Pediatric MRI Sedation

NCT03513757
View directory record Official record
Conditions
Headache, Tumor, Seizure Disorder, Neurofibromatoses, Hydrocephalus, Abdominal Neoplasm, Spine Deformity
Interventions
propofol, Dexmedetomidine, Glycopyrrolate, Lidocaine 1% Injectable Solution, Nitrous Oxide, Sevoflurane
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
12 Months to 60 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 20, 2020 · Synced Jul 25, 2026, 11:23 AM EDT
Completed Phase 4 Interventional Results available

Study to Evaluate Effect of Pandel Cream 0.1% on HPA Axis in Pediatric and Adult Population

NCT01137032
View directory record Official record
Conditions
Psoriasis, Atopic Dermatitis
Interventions
Pandel Cream 0.1%
Drug
Lead sponsor
Fougera Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
3
States / cities
Omaha, Nebraska • Hilton Head Island, South Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated May 3, 2016 · Synced Jul 25, 2026, 11:23 AM EDT